pharmaphorum

OCTOBER 31, 2022

Verge Genomics has joined a select group of biotechs who have taken a drug discovered and developed using artificial intelligence into human testing. The post Verge Genomics takes AI-sourced drug for ALS into clinic appeared first on.

Genomics 136

Genomics Genome Drugs RNA 136

Pharmaceutical Technology

DECEMBER 11, 2022

The US Food and Drug Administration (FDA) has granted Fast Track designation for Pfizer and BioNTech’s messenger ribonucleic acid (mRNA)-based combination vaccine candidate against Covid-19 and influenza. 5 Omicron sublineages spike proteins. The vaccine is intended to prevent two respiratory ailments through a single injection.

Vaccine 289

Vaccine Vaccination Gene Therapy Gene 289

pharmaphorum

MAY 14, 2021

Biogen has made a further push into RNA-based drug discovery via a collaboration with Envisagenics, which applies artificial intelligence RNA sequencing data to discover new drug targets and therapeutics. It’s not the first partnership for Biogen in the RNA splicing area.

RNA 60

RNA Licensing Licensing Genetics 60

pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .

RNA 52

RNA Genetic Disease Drugs Protein 52

Worldwide Clinical Trials

AUGUST 14, 2024

Historically, these indications have challenged the one-size-fits-all treatment approach due to patient variability, such as genetic differences in drug metabolism and underlying health conditions. Protein-based Biomarkers : Include proteins found in blood, tissues, or other bodily fluids that can indicate disease presence or progression.

Genetics 195

Genetics Protein Gene Genotype 195

The Pharma Data

JANUARY 5, 2021

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins. The first identifies the 3D RNA motifs.

RNA 52

RNA Protein Bioavailability DNA 52

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. In three more patients treated at 0.1mg/kg levels fell by 52%.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

pharmaphorum

JANUARY 6, 2022

Roivant is the latest pharma group to take a position in the emerging field of therapies targeted at RNA splicing, licensing exclusive rights to a drug developed by a unit of Japanese drugmaker Eisai. . The post Roivant joins RNA splicing push with Eisai deal appeared first on.

RNA 52

RNA Licensing Licensing Development 52

Pharmaceutical Technology

JULY 5, 2022

Researchers at the UNC School of Medicine have discovered that hepatitis A virus (HAV) replication needs particular interactions between the human protein ZCCHC14 and TENT4 poly(A) polymerases, a group of enzymes. Subsequently, Lemon’s lab tested RG7834 and found the precise effects of the oral drug on HAV in liver and faeces.

Protein 130

Protein RNA Genomics Genome 130

XTalks

AUGUST 5, 2020

In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.

Gene Expression 112

Gene Expression Development RNA Drugs 112

STAT News

AUGUST 17, 2022

On Target is a recurring feature from STAT that dives deep into the most promising drug targets in oncology.    The hunt for cancer cures has, to a large degree, been a hunt for biomarkers — DNA, peptides, RNA, proteins or more — that might set tumor cells apart from healthy tissue.

RNA 98

RNA DNA Protein Scientist 98

Scienmag

NOVEMBER 20, 2020

Scientists at Goethe University within the international consortium COVID19-NMR refine previous 2D models The genetic code of the SARS-CoV2 virus is exactly 29,902 characters long, strung through a long RNA molecule. It contains the information for the production of 27 proteins.

Genome 70

Genome Genomics RNA Protein 70

pharmaphorum

AUGUST 18, 2020

Could AI prevent future pandemics by developing an armoury of drugs that work against all coronaviruses? The consortium aims to rapidly develop drugs to fight SARS-CoV-2 and find virus neutralising antibodies. But also included in the 77.7

Protein 121

Protein Pharma Companies RNA Drugs 121

pharmaphorum

OCTOBER 5, 2022

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018.

RNA 105

RNA Genetics Dermatology Antibody 105

Roots Analysis

OCTOBER 30, 2023

In the past few years, Next Generation RNA therapeutics have emerged as one of the key therapeutic modalities in the modern healthcare industry. These RNA based therapeutics play a crucial role in protein production and regulation of gene functions.

RNA 40

RNA Vaccine Vaccination Development 40

pharmaphorum

APRIL 19, 2022

The funding will be used to continue the clinical development of lead oligonucleotide PGN-EDO51 for DMD, as well as two other oligo drugs PGN-EDODM1 for myotonic dystrophy type 1 (DM1) and PGN-EDO53, another DMD candidate. Once there, they disrupt the expression of RNA coding for disease-associated proteins.

Protein 105

Protein RNA Clinical Development Production 105

pharmaphorum

JANUARY 7, 2022

AstraZeneca has clearly made the rare disease transthyretin amyloidosis (ATTR) a key component of its rare disease pipeline, licensing a second drug candidate in the space of a couple of months. Tafamidis is thought to work by stabilising the misfolded proteins.

Antibody 116

Antibody Licensing Licensing Protein 116

The Pharma Data

MARCH 23, 2022

Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. Drugs that target polymerase could potentially treat Ebola virus infections and save lives. The researchers discovered that Ebola virus polymerase hijacks a cellular protein called GSPT1. Catching polymerase in the act.

Protein 52

Protein Genomics Genome Research 52

XTalks

JANUARY 3, 2024

Wainua is the only FDA-approved drug for the treatment of ATTRv-PN that can be self-administered via an auto-injector. In individuals affected by ATTR, which includes both hereditary and wild-type (non-hereditary) variants, the TTR protein forms fibrils that accumulate in various tissues.

FDA Approval 105

FDA Approval Protein RNA Clinical Trials 105

Delveinsight

JULY 29, 2021

Biogen-Ionis’ experimental drug meets primary goal in Alzheimer’s trial. Biogen and Ionis Pharmaceuticals have demonstrated that their experimental drug, BIIB080 (IONIS-MAPT), fulfilled the primary goal of safety and tolerability in the Phase Ib multiple ascending doses (MAD) clinical trials in mild Alzheimer’s disease patients.

Drugs 98

Drugs Protein Life Science Development 98

pharmaphorum

SEPTEMBER 21, 2020

It is the first and only drug available to treat FCS since being approved by the EMA last year. The drug works by reducing the production of ApoC-III, a protein that regulates plasma triglycerides. The post NICE relents and okays Akcea’s rare disease drug Waylivra for NHS use appeared first on.

Drugs 80

Drugs RNA Medicine Genetics 80

pharmaphorum

AUGUST 10, 2021

Oxford University and UK clinical artificial intelligence (AI) company Sensyne Health will partner on a project to find new drug targets for people with hard-to-treat asthma. Dr Timothy Hinks. According to Hinks, “we still don’t understand the causes of asthma, because in the past the scientific tools just didn’t exist.

Gene Sequencing 116

Gene Sequencing Gene Bioinformatics Genomics 116

Delveinsight

AUGUST 26, 2021

Shape’s RNA editing technologies can modify the RNA sequence, which makes the body’s protein building blocks. This system is designed to deliver RNA editing technology or other payloads directly to particular body areas, such as the nervous system or muscle.

Vaccine 98

Vaccine Vaccination Gene Therapy RNA 98

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. It has a candidate in development that targets TMPRSS6, an enzyme that reduces levels of a protein (hepcidin) that controls iron levels in the body.

Genetic Disease 104

Genetic Disease Protein RNA Doctor 104

pharmaphorum

NOVEMBER 9, 2020

Sanofi’s management has highlighted fitusiran, a potential competitor to Roche’s haemophilia A drug Hemlibra, as one of the most important drugs in its pipeline. Fitusiran is being developed for haemophilia A and B in partnership with RNA interference (RNAi) specialist Alnylam – but has already been affected by safety issues.

RNA 52

RNA Drugs Trials Development 52

pharmaphorum

MARCH 9, 2022

Sanofi and Sobi’s haemophilia partnership has been under competitive pressure from new therapies like Roche’s antibody Hemlibra, so the two companies are hoping a new long-acting drug candidate can revitalise the franchise. The half life of the new drug – one of the main draws for Sanofi’s $11.6

Protein 98

Protein Gene Therapy Sales Regulation 98

pharmaphorum

JANUARY 6, 2021

Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. Danish drugmaker Novo Nordisk signed a deal with the US biotech in 2019 valued at up to $675.5

Gene Silencing 66

Gene Silencing Gene RNA Contract Manufacturing 66

pharmaphorum

JANUARY 12, 2021

US biotech Atalanta Therapeutics has come out of stealth mode backed with $110m from Biogen and Roche, who have also signed separate partnerships to develop new therapies for neurological diseases using RNA interference (RNAi) technology. It also reflects the contribution from Khorova as the founder of the company.

RNA 95

RNA Production Development Pharma Companies 95

pharmaphorum

NOVEMBER 18, 2021

The two companies have been working together since 2019 to develop multiple RNA interference (RNAi) candidates for disorders such as non-alcoholic steatohepatitis (NASH), type 2 diabetes, obesity and rare diseases. Dicerna has been a hot tip for takeover for a couple of years, as the list of approved drugs that work via RNAi has lengthened.

Gene Silencing 52

Gene Silencing RNA Protein Gene 52

XTalks

JANUARY 3, 2025

Ionis Pharmaceuticals recently announced that its drug, Tryngolza (olezarsen), has been approved as an adjunct to diet for the reduction of triglyceride levels in FCS patients, who have long faced a condition with no existing pharmacological solutions. In the Balance study, patients who received the drug showed a 42.5

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

Roots Analysis

MARCH 16, 2023

It is important in the process of protein synthesis because mRNA is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein. In the last few years, researchers have become interested in using in vitro transcribed (IVT) mRNA as a drug delivery agent.

Vaccine 52

Vaccine Vaccination DNA Protein 52

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

Protein 52

Protein RNA Production Genetics 52

Delveinsight

JANUARY 7, 2021

Ikena secures USD 120 Million to advance a growing pipeline of cancer drugs. Ikena Oncology has bagged USD 120 million to develop its expanding pipeline of cancer drugs. The newer side of the pipeline points targeted oncology drugs. Terns raises USD 87 Million for NASH drugs. IND-enabling studies are underway.

RNA 52

RNA Clinical Trials Clinical Trials Bioavailability 52

pharmaphorum

AUGUST 5, 2021

billion upfront to claim ownership of Vividion ‘s drug discovery platform, which uses chemoproteomics to identify new binding sites on proteins and find small-molecule compounds that interact with them, with another $500 million on the table if pipeline candidates meet targets. .” Bayer is paying $1.5 billion ($5.3

DNA 52

DNA Gene Therapy Protein Gene 52

Delveinsight

JANUARY 18, 2021

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome. GeneTx and Ultragenyx are co-developing the therapy.

Marketing 52

Marketing Gene Drugs Protein 52

pharmaphorum

DECEMBER 7, 2021

Roche has formed a decade-long alliance with Recursion Pharma that will focus on using machine learning and “industrialised” drug discovery to find new neuroscience and cancer drugs. The data is fed into mathematical models to find new biological relationships that can be a starting point for discovering new therapeutics.

Drugs 59

Drugs RNA Pharma Companies Protein 59

Roots Analysis

MARCH 8, 2023

Over time, various research studies have demonstrated the potential of exosomes ( membrane bound extracellular vesicles) in disease diagnosis, drug delivery and therapeutic applications. In addition, they are involved in regulating the expression of the targets that are linked to RNA as they can affect the expression of RNA.

RNA 52

RNA Drug Delivery DNA Development 52